Operation Santa to help rare

What does orphan disease means?

Frederick S. Kaplan, MD 1, 2, 4
Robert J. Pignolo, MD, PhD 1, 2, 4
Eileen M. Shore, PhD 1, 3, 4

(From The Departments of 1Orthopaedic Surgery, 2Medicine and 3Genetics and 4The Center for Research in FOP and Related Disorders, The Perelman School of Medicine at the University of Pennsylvania)

The Orphan Drug Act (ODA), enacted by The United States Congress in 1983, encourages pharmaceutical companies to develop drugs for diseases that have a small market. In the US, an orphan drug is one for a disease that affects fewer than 200, 000 individuals. The European Union (EU) has a similar law to promote development of "orphan medicinal products."

The ODA includes provisions to grant special status to products to treat rare diseases. In order to receive Orphan Drug Designation (ODD) for a product, a company sponsor applies to the FDA with a request. If the necessary criteria are met, the ODD is approved, qualifying the sponsor for financial benefits.

ODD provides economic incentives, tax reductions, and the exclusive rights to market a specific compound in the US for a period of seven years after market approval. It encourages companies to enter a market where high costs of drug development are less likely to be recouped quickly, due to the smaller pool of individuals affected.

ODD does not mean that the sponsor has already initiated development of the drug for clinical use; only that they would qualify for incentives under the ODA if they choose to do so. It also does not mean that the drug is safe, effective, or legal to manufacture and market. Standard regulatory processes and requirements are still necessary for obtaining market approval, and safety and efficacy must still be established through clinical studies.

La Jolla Pharmaceutical Company, self-described as "a biopharmaceutical company dedicated to the development of medical treatments that significantly improve outcomes in patients with life-threatening diseases" announced on April 18, 2013 that the FDA Office of Orphan Products Development (OOPD) has granted ODD to La Jolla Pharmaceutical's drug candidate LJPC-6417 (also known as LDN193189) for fibrodysplasia ossificans progressiva (FOP).

LDN193189 was not developed by La Jolla Pharmaceuticals but by scientists at Harvard University and is being studied intensively by the Therapeutics for Rare and Neglected Disease (TRND) program at the NIH. Presently, there is no public knowledge if the Harvard or TRND programs are affiliated with the La Jolla Pharmaceutical filing.

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